Montes Jacqueline, Garber Carol Ewing, Kramer Samantha S, Montgomery Megan J, Dunaway Sally, Kamil-Rosenberg Shirit, Carr Brendan, Cruz Rosangel, Strauss Nancy E, Sproule Douglas, De Vivo Darryl C
Department of Neurology, Columbia University Medical Center, New York, NY, USA.
Department of Rehabilitation and Regenerative Medicine, Columbia University Medical Center, New York, NY, USA.
J Neuromuscul Dis. 2015 Oct 7;2(4):463-470. doi: 10.3233/JND-150101.
The benefits of exercise on long-term health and well-being are well established. The possible benefits of exercise in Spinal Muscular Atrophy (SMA) have not been explored in a controlled clinical trial format.
To assess the effects of exercise on measures of function, strength, and exercise capacity in ambulatory SMA patients.
Fourteen participants, ages 10-48 years, were randomized to control and exercise cohorts after a 1 month lead-in period. The exercise group received 6 months of intervention. Thereafter, both groups received the intervention for the remaining 12 months. Participants were monitored for a total of 19 months. Exercise included individualized home-based cycling and strengthening. The primary outcome measure was distance walked during the six-minute walk test (6MWT). Secondary outcomes included strength, function, exercise capacity, quality of life and fatigue.
Twelve participants completed the first 7 months of the study, and 9 completed all 19 months. At baseline, the groups were similar on all clinical variables. There were no group changes at any time point in the 6MWT, fatigue, or function. Percent-predicted VO2 max improved 4.9% in all participants in 6 months (p = 0.036) (n = 10).
Daily exercise is safe in ambulatory SMA and should be encouraged. We did not uncover any deleterious effects on strength, function, or fatigue. Our study documented a reduction in oxidative capacity and a blunted conditioning response to exercise possibly representing an important insight into underlying pathophysiological mechanisms. These findings also may be linked causally to mitochondrial depletion in SMA and warrant further study.
运动对长期健康和幸福的益处已得到充分证实。运动在脊髓性肌萎缩症（SMA）中的潜在益处尚未在对照临床试验形式中进行探索。
评估运动对非卧床SMA患者功能、力量和运动能力指标的影响。
14名年龄在10 - 48岁的参与者在1个月的导入期后被随机分为对照组和运动组。运动组接受6个月的干预。此后，两组在剩余的12个月中都接受干预。参与者总共被监测19个月。运动包括个体化的家庭骑行和强化训练。主要结局指标是六分钟步行试验（6MWT）期间行走的距离。次要结局包括力量、功能、运动能力、生活质量和疲劳。
12名参与者完成了研究的前7个月，9名完成了全部19个月。在基线时，两组在所有临床变量上相似。在6MWT、疲劳或功能方面，任何时间点两组均无变化。在6个月内，所有参与者预测的最大摄氧量百分比提高了4.9%（p = 0.036）（n = 10）。
日常运动对非卧床SMA患者是安全的，应予以鼓励。我们未发现对力量、功能或疲劳有任何有害影响。我们的研究记录了氧化能力的降低和对运动的适应性反应减弱，这可能代表了对潜在病理生理机制的重要见解。这些发现也可能与SMA中的线粒体耗竭存在因果关系，值得进一步研究。
